Cilostazol for the prevention of acute progressing stroke: A multicenter, randomized controlled trial

Background: Progressing stroke is one of the major determinants of outcome after acute ischemic stroke. A pilot randomized controlled trial was conducted to investigate the effect of cilostazol on progressing stroke. Methods: Adult patients with noncardioembolic ischemic stroke within 24 hours after onset were randomized to receive cilostazol 200 mg/day (cilostazol group) or no medication (control group) in addition to the optimum medical treatments (a free radical scavenger plus an antiplatelet agent or an antithrombin agent). The primary endpoints were the rate of progressing stroke, defined as aggravation of the National Institutes of Health Stroke Scale (NIHSS) score by ≥4 points on days 3 and/or 5 and a modified Rankin Scale score of 0 to 1 at 3 months after enrollment. Aggravation caused by systemic complications, edema, hemorrhagic infarction, or recurrent stroke was not considered as progressing stroke. This trial was registered as UMIN000001630. Results: A total of 510 patients were enrolled from 55 institutions in Japan between February 2009 and July 2010. The rate of progressing stroke was 3.2% and 6.3% in the cilostazol and control groups, respectively (P =.143). The modified Rankin Scale score of 0 to 1 at 3 months did not differ between the groups. Conclusions: Cilostazol failed to show a preventive effect against acute progressing stroke. However, the tendency to reduce progressing stroke and the results of stratified analyses may encourage additional studies to clarify the effect of cilostazol in the treatment of acute ischemic stroke.